With the aim of improving the lives of all those affected by a rare disease, the United Kingdom Government published The UK Strategy for Rare Diseases in 2013, a high-level framework containing 51 commitments which sets out a seven-year strategic vision from 2013-2020. A copy of the Strategy is attached. The Government is committed to implementing the Strategy’s commitments and has, in January 2018, published two implementation plans setting out its actions for England. Copies of the Department’s The UK Strategy for Rare Diseases: Rare Diseases implementation plan for England and NHS England’s Implementation Plan for the UK Strategy for Rare Diseases are attached.
The Government has not made any assessment of differences in access to medicines for the treatment of rare diseases or on the available number of treatments between England, the devolved administrations and economically similar countries in Europe.
With regard to the Government’s assessment of the sustainability of the National Institute for Health and Care Excellence’s (NICE) appraisal process for rare disease medicines that do not meet the highly specialised technology criteria, patients with rare diseases in England benefit from the same NHS Constitution right to clinically and cost-effective medicines as patients with more common conditions. NICE has recommended a number of medicines for the treatment of rare diseases through its technology appraisal and highly specialised technology evaluation programmes which are now routinely available to National Health Service patients in line with NICE’s recommendations.